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Cellectar's Iopofosine I 131 Doubles Survival in Pediatric Brain Cancer Trial

Rare DiseaseOrphan Drug
  • Cellectar Biosciences reported that iopofosine I 131 achieved 5.4 months average progression-free survival in pediatric high-grade glioma patients, doubling the reported median of 2.25 months.
  • Patients receiving higher doses (minimum 55 mCi) showed 8.6 months overall survival, with three patients receiving additional cycles achieving 11.5 months average overall survival.
  • The radioligand therapy demonstrated a favorable safety profile with no cardiovascular, renal, or liver toxicities, and all hematologic adverse events were manageable and reversible.
  • The CLOVER-2 Phase 1 trial enrolled 14 pediatric patients with relapsed/refractory high-grade gliomas, including diffuse midline gliomas and diffuse intrinsic pontine gliomas.

Novel Targeted Radiotherapy in Pediatric Patients With Inoperable Relapsed or Refractory HGG

NCT05610891Active, Not RecruitingPhase 1
Cellectar Biosciences, Inc.
Posted 10/1/2023

Study to Compare Tivozanib in Combination With Nivolumab to Tivozanib Monotherapy in Subjects With Renal Cell Carcinoma

NCT04987203Active, Not RecruitingPhase 3
AVEO Pharmaceuticals, Inc.
Posted 9/9/2021

Dose Escalation Study of CLR 131 in Children, Adolescents, and Young Adults With Relapsed or Refractory Malignant Tumors Including But Not Limited to Neuroblastoma, Rhabdomyosarcoma, Ewings Sarcoma, and Osteosarcoma

NCT03478462Active, Not RecruitingPhase 1
Cellectar Biosciences, Inc.
Posted 4/30/2019

SpliceBio Raises $135 Million Series B to Advance First Dual AAV Gene Therapy for Stargardt Disease

Orphan Drug
  • SpliceBio secured $135 million in Series B funding co-led by EQT Life Sciences and Sanofi Ventures to advance SB-007, the first dual AAV gene therapy cleared by FDA for Stargardt disease treatment.
  • The company's proprietary Protein Splicing platform addresses a fundamental limitation of AAV vectors by enabling delivery of large genes that exceed the 4.7 kilobase packaging capacity through engineered inteins.
  • SB-007 has received Orphan Drug Designation from both FDA and European Commission, with the first patient dosed in the Phase 1/2 ASTRA study in March 2025.
  • The platform technology has transformative potential across ophthalmology and neurology by unlocking treatment possibilities for genetic disorders previously considered untreatable due to large gene size limitations.

Daewoong Pharmaceutical Partners with Salipro Biotech to Target Challenging Membrane Proteins for Drug Discovery

Drug DiscoveryOrphan Drug
  • Daewoong Pharmaceutical has entered into a strategic research collaboration with Swedish biotech Salipro Biotech to develop novel small molecule therapeutics targeting challenging membrane proteins.
  • The partnership provides Daewoong access to Salipro's proprietary platform technology that stabilizes membrane proteins in their native forms, addressing a major barrier in drug discovery.
  • This marks Salipro's first collaboration with a Korean company, following previous partnerships with major pharmaceutical firms including Boehringer Ingelheim, Sanofi, and Sumitomo.
  • Membrane proteins account for over 60 percent of known drug targets but have been difficult to research due to their structural instability in laboratory conditions.

Hemab Therapeutics Advances Bleeding Disorder Pipeline with Promising Clinical Data at ISTH 2025

Monoclonal AntibodyOrphan Drug
  • Sutacimig (formerly HMB-001) demonstrates promising interim Phase 2 safety and efficacy results for Glanzmann thrombasthenia, receiving WHO International Non-Proprietary Name designation.
  • HMB-002 shows initial proof of mechanism clinical data in Von Willebrand disease patients through the ongoing VELORA Pioneer study, supported by comprehensive preclinical data.
  • Natural history studies reveal significant disease burden, with 88% of Glanzmann thrombasthenia patients reporting at least one bleed weekly and 34% requiring medical treatment.
  • Hemab will present 11 abstracts across multiple bleeding disorder programs at the International Society on Thrombosis and Haemostasis Congress in Washington, DC.

A Study of Bleeding and Treatment in Participants With Von Willebrand Disease

NCT06610201Recruiting
Hemab ApS
Posted 8/30/2024

A Study Assessing HMB-002 in Participants With Von Willebrand Disease

NCT06754852RecruitingPhase 1
Hemab ApS
Posted 2/6/2025

A Phase 1/2 Study to Investigate Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of HMB-001 in Glanzmann Thrombasthenia

NCT06211634Active, Not RecruitingPhase 1
Hemab ApS
Posted 12/13/2022

SIFI Merges with Faes Farma to Expand Global Access to Akantior, First Approved Therapy for Rare Eye Infection

Orphan DrugRare DiseaseDrug Approval
  • SIFI, a leading international ophthalmic company, has agreed to merge with European pharmaceutical company Faes Farma in a transaction that positions ophthalmology as approximately 20% of the combined entity's pro-forma revenue.
  • The merger will accelerate development of Akantior (polihexanide 0.08%), the first and only approved therapy worldwide for Acanthamoeba Keratitis, particularly targeting expansion into the US market where it has orphan drug designation.
  • The combined entity will leverage commercial synergies across complementary markets in Europe and Latin America, while also advancing polihexanide development for additional orphan indications including Fungal Keratitis.
  • The transaction values SIFI at a double-digit multiple of 2024 EBITDA plus regulatory and commercial milestone-based earn-outs, with closing expected in Q3 2025 subject to regulatory approvals.

Citius Oncology Partners with Cardinal Health for LYMPHIR Distribution Ahead of Commercial Launch

Targeted TherapyDrug ApprovalOrphan Drug
  • Citius Oncology has entered into a distribution services agreement with Cardinal Health to support the upcoming U.S. commercial launch of LYMPHIR for cutaneous T-cell lymphoma treatment.
  • LYMPHIR is an FDA-approved targeted immunotherapy indicated for adults with relapsed or refractory Stage I-III cutaneous T-cell lymphoma after at least one prior systemic therapy.
  • The partnership leverages Cardinal Health's proven specialty pharmaceutical distribution capabilities to ensure efficient and reliable access to LYMPHIR for healthcare providers and patients.
  • Management estimates the initial market for LYMPHIR currently exceeds $400 million and is underserved by existing therapies.

Avanzanite Partners with Agios to Commercialize First-in-Class Pyruvate Kinase Activator Across Europe

Rare DiseaseDrug ApprovalOrphan Drug
  • Avanzanite Bioscience has secured exclusive European commercialization rights for PYRUKYND® (mitapivat), a first-in-class pyruvate kinase activator approved for treating adults with PK deficiency.
  • The partnership covers distribution across the European Economic Area, UK, and Switzerland, addressing an ultra-rare inherited condition that previously had no approved treatments.
  • Avanzanite plans to expand into 32 European countries over the next 12 months, having already tripled its Q1 2025 revenue year-over-year with two rare disease medicines currently on market.

Four Promising Gene and Cell Therapies Challenge Aldurazyme's Dominance in MPS I Treatment

Orphan DrugRare Disease
  • Four innovative therapies are advancing through clinical trials to address critical unmet needs in MPS I treatment, particularly targeting central nervous system complications that current therapy Aldurazyme cannot reach.
  • Orchard Therapeutics' OTL-203 gene therapy is progressing through Phase III trials with planned US submission in 2028, while JCR Pharmaceuticals' JR-171 offers blood-brain barrier penetration capabilities.
  • REGENXBIO's RGX-111 gene therapy recently secured a strategic partnership with Nippon Shinyaku for development in the US and Asia, and Immusoft's ISP-001 cell therapy reported encouraging Phase I results.
  • These next-generation treatments aim to provide superior neurological efficacy and patient convenience compared to the current standard requiring lifelong weekly infusions.

FDA Rejects Elamipretide for Rare Mitochondrial Disease Despite Patient Improvements

Rare DiseaseOrphan Drug
  • The FDA's Division of Cardiology and Nephrology rejected elamipretide's new drug application on May 29, 2025, despite reported improvements in patients with rare mitochondrial diseases.
  • Four-year-old Hope Filchak with Microphthalmia with Linear Skin Defects syndrome showed stabilized heart function and increased energy after 18 months of elamipretide treatment.
  • Stealth Biotherapeutics has reduced overhead by 30% and estimates it can continue manufacturing for only six to nine months without FDA approval.
  • The FDA's rejection was partly based on lack of improvement in six-minute walk tests, which experts argue is not an appropriate outcome measure for mitochondrial diseases.

CNS Pharmaceuticals Presents Brain-Penetrating Taxane TPI 287 for Glioblastoma Treatment at 2025 Brain Tumor Summit

Orphan Drug
  • CNS Pharmaceuticals' Chief Medical Officer Sandra Silberman presented TPI 287, a brain-penetrating abeotaxane, at the 2025 Brain Tumor Biotech Summit on June 5, 2025.
  • TPI 287 demonstrated promising efficacy in a Phase 1 trial for glioblastoma, achieving 3 complete responses and 9 partial responses out of 23 evaluable patients when combined with bevacizumab.
  • The drug has received FDA Orphan Drug Designation for treating gliomas, pediatric neuroblastoma, and progressive supranuclear palsy, distinguishing it from other taxanes that cannot cross the blood-brain barrier.
  • TPI 287 has been tested in over 350 patients across multiple clinical trials, showing an excellent safety profile and high tolerability for various brain and central nervous system cancers.

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